Childhood-onset striatonigral degeneration is a rare genetic disorder that robs children of the ability to walk and speak by ...

Upon close of merger and $78.9mm concurrent private placement from a syndicate of leading healthcare-dedicated investors, ...

Thailand’s Defence Ministry added three unusual medical conditions to the exemption list for compulsory military service.

Matilda was diagnosed with a very rare syndrome but her parents 'want her to be able to go out in the world and do what she ...

Recent research has revealed that nitisinone, a drug originally used to treat rare genetic conditions, can fatally disrupt a ...

Immunodeficiency disorders linked to cytoskeleton defects are rare and complex, often presenting with diverse clinical ...

A football legend has sent an 11-year-old non verbal boy who is flying out to Mexico for treatment a video message of hope.

But her career arc slowly bent back toward that childhood revelation. Today, Stueland is the CEO of GeneDx, a publicly traded ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, granted equity awards ...

Investing.com -- Moderna (NASDAQ: MRNA) shares dropped 13% pre-open on Monday as investors reacted to the weekend news that the U.S. Food and Drug Administration’s (FDA) top vaccine official, Dr.

Marks’ resignation leaves the field without a regulator many view as “integral” to its progress over the last decade.

Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage biopharmaceutical company focused on developing innovative treatments for patients with unmet medical needs, today announced fourth quarter ...